UPDATED: Intercept Pharma gets accelerated FDA OK for liver drug Ocaliva

Intercept Pharmaceuticals ($ICPT) has successfully wrapped up its quest for an accelerated approval of its would-be blockbuster obeticholic acid for primary biliary cirrhosis (PBC). The New York-based biotech put out the word late Friday that the FDA had come through with an expected green light for marketing the drug.

The early approval for the drug in combination with ursodeoxycholic acid was widely expected following a mildly critical internal review at the agency and a unanimous endorsement from an agency advisory panel.

The drug will be marketed as Ocaliva, with plans to hit the market in the next 7 to 10 days. On Tuesday morning the company told analysts that the drug will sell for $69,350 a year. But after watching its shares surge in premarket trading on Tuesday, the stock price slipped into the red by midmorning.

Intercept noted in its midnight release that this is the first new drug for PBC in close to 20 years.

"Ocaliva fills an important unmet need for the many patients with PBC who have an inadequate response to or are intolerant of UDCA, which until now has been the only approved treatment," said John Vierling, a professor at Baylor College of Medicine, in a statement. "Ocaliva has demonstrated a clinically meaningful improvement in lowering ALP, a liver enzyme and biomarker that is used to track disease progression in patients with PBC. Importantly, Ocaliva maintained durable ALP reductions, which is critical for treatment of a chronic disease like PBC."

The FDA advisers felt that data on a single biomarker for the disease--using alkaline phosphatase (ALP) as a surrogate endpoint for transplant-free survival--should be enough to warrant an approval. Data on another key biomarker, total bilirubin--which could come from a confirmatory study--were largely absent in the biotech’s NDA. 

The panel also backed starting the dose at 5 mg and then titrating up to 10 mg in order to achieve a response while guarding against side effects. That point was picked up by the agency, which included the dosing regimen in the drug label. And many felt that there are enough data to support use of OCA as a monotherapy for a small number of patients who can't tolerate ursodeoxycholic acid.

Analysts have high hopes for this drug. While PBC is the first market Intercept will tackle, some analysts have been absolutely giddy at the prospect of seeing Ocaliva go on to be approved to treat NASH, or fatty liver disease, which could open up a much bigger market worth billions.

“Through over 60 discussions with payers, management is confident that OCA will launch with some commercial coverage, but that P&T meetings may take a few months, with Medicare taking up to 180 days,” noted Baird’s Brian Skorney recently. “The first material U.S. PBC sales are expected in 3Q16, and not until 2017 in Europe, post a potential late 2016 EMA approval.”

The company is planning a call with analysts on Tuesday morning.

- here's the release

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